A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Malnutrition, both moderate and severe, is fairly common among children with epidermolysis bullosa (EB) living in India, a study suggests. A child’s degree of malnutrition also correlated with disease severity, its researchers reported. A personalized diet followed for six months significantly improved patients’ nutritional status, particularly among children with dystrophic…

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…